2 results
Approved WMORecruiting
the objective of the current study is to evaluate the safety and efficacy of enzyme therapy in the Dutch population of patients with MPS I, II and VI; to compare this to the natural course of these diseases; to make an inventory of the direct and…
Approved WMOCompleted
Primary: To assess the safety profile of selnoflast compared with that of placeboSecondary: - To evaluate the efficacy of selnoflast compared with that of placebo- To assess the pharmacokinetic properties of selnoflast and metabolite(s) as…